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Alglucosidase Alfa Temporary Access Program (ATAP)

This treatment has been approved for sale to the public.
Information provided by (Responsible Party):
Sanofi ( Genzyme, a Sanofi Company ) Identifier:
First received: August 21, 2007
Last updated: February 4, 2014
Last verified: February 2014
Pompe disease (also known as glycogen storage disease Type II) is caused by a deficiency of a critical enzyme in the body called acid alpha-glucosidase (GAA). Normally, GAA is used by the body's cells to break down glycogen (a stored form of sugar) within specialized structures called lysosomes. In patients with Pompe disease, an excessive amount of glycogen accumulates and is stored in various tissues, especially heart and skeletal muscle, which prevents their normal function. The objective of this expanded access study is to provide patients with Pompe disease in the United States (US), access to alglucosidase alfa produced from a scaled up manufacturing process for a limited time until production at this scale is approved for commercial use by the Food and Drug Administration.

Condition Intervention
Glycogen Storage Disease Type II (GSD-II) Pompe Disease (Late-Onset) Acid Maltase Deficiency Disease Glycogenosis 2 Biological: alglucosidase alfa (recombinant human acid alpha-glucosidase [rhGAA])

Study Type: Expanded Access     What is Expanded Access?
Official Title: Alglucosidase Alfa Temporary Access Program

Resource links provided by NLM:

Further study details as provided by Sanofi ( Genzyme, a Sanofi Company ):

Intervention Details:
    Biological: alglucosidase alfa (recombinant human acid alpha-glucosidase [rhGAA])
    IV infusion: 20mg/kg qow
    Other Name: Alglucosidase alfa

Ages Eligible for Study:   18 Years and older   (Adult, Senior)
Sexes Eligible for Study:   All

Inclusion Criteria:

  • The patient or patient's legal guardian must provide signed, informed consent prior to performing any study-related procedures.
  • The patient must reside in the US.
  • The patient must have a confirmed diagnosis of Pompe disease defined as documented acid alpha-glucosidase (GAA) enzyme deficiency from any tissue source and/or GAA gene mutations.
  • The patient must have/had documented clinical signs and symptoms of Pompe disease.
  • The patient must have/had prior treatment with alglucosidase alfa produced at commercial scale OR be naive to enzyme replacement therapy (ERT) for the treatment of Pompe disease and meet at least 1 of the following criteria: require a wheelchair OR require some respiratory assistance for any number of hours (including night time) through non-invasive ventilation.
  • The patient must be capable of complying with the required program schedule of assessments.

Exclusion Criteria:

  • Females who are pregnant or lactating
  • The patient has a clinical condition unrelated to Pompe disease that would interfere with program assessments.
  • The patient is currently enrolled in any clinical studies.
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its identifier: NCT00520143

  Show 74 Study Locations
Sponsors and Collaborators
Genzyme, a Sanofi Company
Study Director: Medical Monitor Genzyme, a Sanofi Company
  More Information

Responsible Party: Genzyme, a Sanofi Company Identifier: NCT00520143     History of Changes
Other Study ID Numbers: AGLU03907
Study First Received: August 21, 2007
Last Updated: February 4, 2014

Keywords provided by Sanofi ( Genzyme, a Sanofi Company ):
GSD-II, Pompe Disease, Glycogen Storage Disease II

Additional relevant MeSH terms:
Glycogen Storage Disease Type II
Glycogen Storage Disease
Deficiency Diseases
Lysosomal Storage Diseases, Nervous System
Brain Diseases, Metabolic, Inborn
Brain Diseases, Metabolic
Brain Diseases
Central Nervous System Diseases
Nervous System Diseases
Metabolism, Inborn Errors
Genetic Diseases, Inborn
Carbohydrate Metabolism, Inborn Errors
Lysosomal Storage Diseases
Metabolic Diseases
Nutrition Disorders processed this record on August 23, 2017